Ionis Pharmaceuticals' Rare Metabolic Disorder Candidate Poised To Become New Standard Treatment, Study Shows Encouraging Action

Ionis Pharmaceuticals Inc IONS released topline results for the Phase 3 Balance study of olezarsen in people with familial chylomicronemia syndrome (FCS), a hereditary disease that prevents the body from breaking down fats.

The trial met its primary efficacy endpoint with a statistically significant reduction in triglyceride (TG) levels with the olezarsen 80 mg monthly dose at six months compared to placebo (p=0.0009); triglyceride lowering continued to improve at 12 months. 

Also Read: Analyst Upgrades Ionis Pharmaceuticals To Buy Amid Key Pipeline Developments: Eplontersen And Donidalorsen Show Promising Outlook

In addition, olezarsen 80 mg showed a 100% reduction in acute pancreatitis events compared to placebo (0 events for olezarsen versus 11 events for placebo), a key secondary endpoint.

Treatment with olezarsen 80 mg resulted in a >75% reduction in apoC-III, a protein produced in the liver that regulates TG metabolism in the blood. 

In addition to the 80 mg monthly dose, the study also evaluated a lower 50 mg monthly dose. Olezarsen demonstrated a dose-dependent effect, with both study doses showing a substantial reduction in pancreatitis. 

The lower 50 mg dose did not reach statistical significance at six months on the primary endpoint of triglyceride-lowering (p=0.0775).

Ionis plans to file a New Drug Application in early 2024 with the FDA.

Olezarsen demonstrated a favorable safety and tolerability profile in the study. 

Price Action: IONS shares are up 7.83% at $46.00 on the last check Tuesday.